Outcomes of pediatric liver transplantation for progressive familial intrahepatic cholestasis.

BACKGROUND: Progressive familial intrahepatic cholestasis (PFIC) is a heterogenous group of inherited hepatocellular disorders and the clinical aspects, role of liver transplantation (LT), and its outcomes remain largely unelucidated. We present our data of LT for each type of PFIC and compare their early, and long-term outcomes, highlighting their individual differences and management strategies. METHODS: Prospectively collected data over a decade (2011-2022) of children with PFIC who underwent LT was analyzed. The groups (PFIC 1-4) were compared with regard to early and long-term outcomes including attainment of catch-up growth. RESULTS: Of 60 children with PFIC who underwent LT, 13, 11, 31 & 5 were of PFIC 1, 2, 3 & 4, respectively. There were no significant differences in gender, PELD scores, BMI, type of grafts, cold and warm ischemia times, intraoperative blood loss, and morbidity among the groups. Post-LT chronic diarrhea was observed in 6 (46.1%) children with PFIC-I, and of them, 3 (23%) developed graft steatohepatitis. Three of these children underwent total internal biliary diversion (TIBD) and on 1-year follow-up, their graft steatosis resolved and they attained catch-up growth. Catch-up growth was significantly poorer in the PFIC1 group (44.4% vs. 88%, 90%, 100% p < .001). Overall 1- and 5-year patient survival of the four PFIC groups (1-4) were 69.2%, 81.8%, 96.8%, 100% & 69.2%, 81.8%, 96.8%, 100%, respectively. CONCLUSION: Ours is the largest to-date series of LT for PFIC illustrating their short- and long-term outcomes. While the results for the whole cohort were excellent, those after LT for PFIC1 was relatively poorer as reflected by catch-up growth, graft steatosis, and post-LT diarrhea, which can be optimized by the addition of TIBD during LT.

Non-standard hepatic venous reconstruction in the setting of absent inferior vena cava in partial graft pediatric liver transplantation-A matched retrospective cohort study.

Recipient cava may be unavailable for outflow reconstruction in some children undergoing liver transplantation (PLT) due to caval agenesis, tumor, or fibrotic caval occlusion. Non-standard hepatic venous reconstruction (NHVR) with a direct veno-caval anastomosis or neo-cava reconstruction is necessary in such cases. Retrospective review of all PLT needing NHVR performed in our unit from January 2010 to September 2019 was performed. Outcomes of this group were compared to a 2:1 matched control group who underwent transplantation with standard piggyback technique. Fifteen children (4.9%) of 304 PLT recipients underwent NHVR. Caval agenesis in biliary atresia (n = 5, 33%) and hepatoblastoma infiltrating the cava (n = 4, 27%) were the commonest indications. Ten children had neo-cava reconstruction, while 5 had direct anastomosis to the supra-hepatic caval cuff or right atrium. One child had developed neo-cava thrombosis without graft venous outflow obstruction in the post-operative period. There was no significant difference in major morbidity, need for re-operation (20% vs 16.7%; P = 1.00), hospital stay (24 days, vs 21 days; P = .32), graft & patient survival among the study and control groups. Absent or inadequate recipient cava during PLT with a partial liver graft can be safely managed with technical modifications. Results equivalent to standard piggyback implantation can be achieved.